Results from Expanded Access Programs: A Review of Academic Literature.

ABSTRACT:

Background

Although expanded access is an increasingly used pathway for patients to access investigational medicine, little is known on the magnitude and content of published scientific research collected via expanded access.

Methods

We performed a review of all peer-reviewed expanded access publications between January 1, 2000 and January 1, 2022. We analyzed the publications for drugs, diseases, disease area, patient numbers, time, geographical location, subject, and research methodology (single center/multicenter, international/national, prospective/retrospective). We additionally analyzed endpoints reported in all COVID-19-related expanded access publications.

Results

We screened 3810 articles and included 1231, describing 523 drugs for 354 diseases for 507,481 pa

SUBMITTER: Polak TB 

PROVIDER: S-EPMC10193319 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature